2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic infusions of factor IX This trial found that a wildtype factor IX gene therapy reduced bleeding - and the burden of infusions https://t.co/exkrInT3BF https://t.co/m5hFS8fNVc
Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B.
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Sep 2024 – Nov 2024Where it's discussed
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News72 articles
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X / Twitter46 posts
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Blogs6 articles
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Facebook3 posts
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YouTube2 videos
AI synopsis
What people are saying
Overall, the discussions demonstrate strong consensus on the therapy's promise, with comments citing specific trial outcomes such as a 71% reduction in bleeding rates and factor IX activity levels of around 27%, while some posts debate costs and logistical challenges, reflecting both excitement and critical analysis.
Most discussions recognize the positive outcomes of the gene therapy, emphasizing significant reductions in bleeding and support for the research findings.
Posts show high interest, with many referencing trial results, long-term benefits, and clinical implications, indicating engagement with the topic.
Some discussions delve into specifics like factor IX levels and side effects, suggesting moderate depth of engagement, though a few comments are more superficial.
The consensus highlights the potential of this therapy to transform treatment for hemophilia B, stressing its significance as a safer and less burdensome alternative.
Abstract in brief
- Fidanacogene elaparvovec is a genetic therapy for hemophilia B that showed promising results in a phase 1-2a study, maintaining high levels of factor IX activity.*
- In a phase 3 study involving men aged 18 to 65 with severe hemophilia B, the therapy led to a significant 71% reduction in bleeding episodes over 15 months compared to standard prophylactic treatment.*
- Despite some participants needing glucocorticoids for side effects, the therapy was generally safe, with no serious adverse infusion events reported.*
Top posts
Most liked on X and BlueskyThe Pfizer #hemophilia B gene therapy 15 month results are in the current issue of the @NEJM. This is AAV Padua IX at 5e11. 15 month mean IX was 27% (median 23%). 62% received steroids! Paper is behind a paywall. https://t.co/v8TYlsoJGr https://t.co/lHy9XmVNV2
Original Article: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B (BENEGENE-2) https://t.co/Lg0GW3rzqZ #Hematology https://t.co/l6xJdTcrhF
In participants with hemophilia B, fidanacogene elaparvovec was superior to factor IX prophylaxis, with a lower bleeding rate and stable factor IX expression. Read the full BENEGENE-2 trial results: https://t.co/Lg0GW3rzqZ https://t.co/c3kfs1x2G6
The @broomedocs podcast up in video format, although why anyone would want to see us (let alone hear us) is beyond me I have have some learning to do for future edits (the AI edit is rather jumpy), but it's a start #FOAMed https://t.co/3SAey1VP3e
Gene Therapy with #Fidanacogene Elaparvovec in Adults with Hemophilia B https://t.co/IEnUKpeQA6
All mentions
Gene Therapy for Hemophilia B Shows Promising Results in Clinical Trials
Fidanacogene elaparvovec, a genetic therapy for hemophilia B, maintained high factor IX levels in early trials and reduced bleeding episodes by 71% over 15 months in a phase 3 study. The treatment was generally safe…
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Phase 3 open-label study, fidanacogene elaparvovec, a gene therapy for hemophilia B, was evaluated in 45 men with severe hemophilia B. The treatment significantly reduced the annualized bleeding rate by 71%. https://t.co/hjTAdGQrr8 Supported by: https://t.co/OwnaMKirrE
RT @First10EM: The @broomedocs podcast up in video format, although why anyone would want to see us (let alone hear us) is beyond me I have…
RT @First10EM: The @broomedocs podcast up in video format, although why anyone would want to see us (let alone hear us) is beyond me I have…
RT @First10EM: The @broomedocs podcast up in video format, although why anyone would want to see us (let alone hear us) is beyond me I have…
RT @First10EM: The @broomedocs podcast up in video format, although why anyone would want to see us (let alone hear us) is beyond me I have…
The @broomedocs podcast up in video format, although why anyone would want to see us (let alone hear us) is beyond me I have have some learning to do for future edits (the AI edit is rather jumpy), but it's a start #FOAMed https://t.co/3SAey1VP3e
Promising results from the Fidanacogene Elaparvovec gene therapy trial in Hemophilia B suggest potential long-term cost-effectiveness over standard treatments, despite current concerns about high costs. https://t.co/4Xn1i3obcB
Hemofili B'de gen tedavisi yapıldı. Virus vektörü aracılığıyla (Fidanacogene Elaparvovec) gen tedavisi Hemofili B tedavisinde etkili. Bu Hemofili B için kullanılan 2. etkili gen tedavisi oluyor. https://t.co/MW8zt9SBBp
Hemofili B'de gen tedavisi yapıldı. Virus vektörü aracılığıyla (Fidanacogene Elaparvovec) gen tedavisi Hemofili B tedavisinde etkili. Bu Hemofili B için kullanılan 2. etkili gen tedavisi oluyor. https://t.co/nIAEgfgUua
RT @wfhemophilia: A study of fidanacogene elaparvovec #GeneTherapy for #hemophilia B demonstrated a 71% reduction in annualized bleeding ra…
A study of fidanacogene elaparvovec #GeneTherapy for #hemophilia B demonstrated a 71% reduction in annualized bleeding rates & factor IX levels of 26.9% at 15months following infusion. 16% of the 46 participants reported serious adverse events. Read more➡️ https://t.co/ToXfQl5Hk3 https://t.co/0a6HLs6Nj1
View a new hemophilia publication in the NEJM that describes the prospective outcomes of gene therapy as a therapeutic approach for adults diagnosed with #HemophiliaB. Click the link below to learn more: https://t.co/JgEolPSCwi https://t.co/wMybxUyZCN
In @NEJM, before/after trial of gene therapy for haem B using fidanacogene elaparvovec. n=45, 188 ineligible due to AAV neutralising Abs. Patients had to have been on >6 m factor prophylaxis. Mean FIX level at 15m: 27% & annualised bleeding rate 4.4 ➡️ 1.3 https://t.co/YG0OKmMKaI https://t.co/Nd2fjE7x24
RT @2minmed: #VisualAbstract: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B is Superior to Prophylaxis https://t.c…
@robertopaulos85 @HorseRenoir83 @HeyNurseKat @_johnbye @Jimmy78591 Yes mate. Virology is certainly on its last legs. You'd best go and tell the people currently using viruses therapeutically. I'm sure they'll bow to the superiority of a random Internet tit with Del Boy as his profile picture.
#VisualAbstract: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B is Superior to Prophylaxis https://t.co/0WG37Lhplq #StudyGraphics #factorix https://t.co/DtlvWFg6Fg
Trend up+6: Cuker A, ..., Benegene-2 Trial Investigators (N Engl J Med) __Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B__ https://t.co/mvOnPzlQCB
RT @NEJM: Original Article: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B (BENEGENE-2) https://t.co/Lg0GW3rzqZ #…
RT @NEJM: Original Article: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B (BENEGENE-2) https://t.co/Lg0GW3rzqZ #…
RT @NEJM: Original Article: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B (BENEGENE-2) https://t.co/Lg0GW3rzqZ #…
RT @NEJM: Original Article: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B (BENEGENE-2) https://t.co/Lg0GW3rzqZ #…
Original Article: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B (BENEGENE-2) https://t.co/Lg0GW3rzqZ #Hematology https://t.co/l6xJdTcrhF
RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…
RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…
RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…
RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…
RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…
RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…
RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…
RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…
2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic infusions of factor IX This trial found that a wildtype factor IX gene therapy reduced bleeding - and the burden of infusions https://t.co/exkrInT3BF https://t.co/m5hFS8fNVc
Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B #science #publication #research #publications https://t.co/3rJT836r80
RT @flora_peyvandi: Gene Therapy with #Fidanacogene Elaparvovec in Adults with Hemophilia B https://t.co/IEnUKpeQA6
RT @ProfMakris: The Pfizer #hemophilia B gene therapy 15 month results are in the current issue of the @NEJM. This is AAV Padua IX at 5e11…
RT @ProfMakris: The Pfizer #hemophilia B gene therapy 15 month results are in the current issue of the @NEJM. This is AAV Padua IX at 5e11…
Gene Therapy with #Fidanacogene Elaparvovec in Adults with Hemophilia B https://t.co/IEnUKpeQA6
RT @ProfMakris: The Pfizer #hemophilia B gene therapy 15 month results are in the current issue of the @NEJM. This is AAV Padua IX at 5e11…
RT @ProfMakris: The Pfizer #hemophilia B gene therapy 15 month results are in the current issue of the @NEJM. This is AAV Padua IX at 5e11…
The Pfizer #hemophilia B gene therapy 15 month results are in the current issue of the @NEJM. This is AAV Padua IX at 5e11. 15 month mean IX was 27% (median 23%). 62% received steroids! Paper is behind a paywall. https://t.co/v8TYlsoJGr https://t.co/lHy9XmVNV2
Hemophilia B gene therapy paper on $PFE's Fidanacogene Elaparvovec published in @nejm: https://t.co/5sFR8fYQhp Challenges remaining for field (in order of importance): 1) huge inter-patient variability in Factor IX expression 2) 56% of patients were administered prolonged https://t.co/6fOqHCcOIe
Het moge duidelijk zijn dat deze gentherapie veel minder belastend is voor patienten dan de huidige standaard, die regelmatig infusies vereist. Voor velen betekent deze nieuwe therapie een leven zonder voortdurende zorg. De hele studie is hier te lezen https://t.co/YFMikMRMfN
Fidanacogene elaparvovec, un vector de terapia génica del virus adenoasociado (AAV) para la hemofilia B que contiene una variante del factor IX humano de alta actividad (FIX-R338L/FIX-Padua), se asoció con una actividad sostenida del factor IX en fase 1-2a https://t.co/o2LcNsnngw
Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B https://t.co/Ce4Md5mJYb https://t.co/HbHt2BpXHq
Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B https://t.co/5zpmQWhaGl
RT @NEJM: In participants with hemophilia B, fidanacogene elaparvovec was superior to factor IX prophylaxis, with a lower bleeding rate and…
In participants with hemophilia B, fidanacogene elaparvovec was superior to factor IX prophylaxis, with a lower bleeding rate and stable factor IX expression. Read the full BENEGENE-2 trial results: https://t.co/Lg0GW3rzqZ https://t.co/c3kfs1x2G6